Retrotope To Present First Human Data On Safety And Early Efficacy Of RT001 With Neurodegeneration At American Academy of Neurology Meeting

LOS ALTOS, CA, April 21, 2017 – Dr. Theresa Zesiewicz, University of South Florida Movement Disorders Clinic, will give a podium presentation at the American Academy of Neurology annual meeting of clinical trial results of Retrotope’s RT001 in the neuromuscular disease, Friedreich’s ataxia (FA) on April 24, 2017.

The study showed good safety and tolerability of this novel drug class, including early signals of efficacy with regard to disease progression. The trial, a randomized, double-­-blind, comparator-controlled study of RT001 in 18 FA patients for 28 days, met all of its primary safety, tolerability and pharmacodynamics (PK) goals. While biological activity was not a primary goal of the study, a number of clinically important disease progression measures showed signals of drug effect in post hoc analysis, unexpected in such a short, small study.

Related news: Retrotope Announces Phase I/II Clinical Trial Results of RT001 in Treatment of Friedreich's Ataxia